Latest news with #Napo
Miami Herald
14 hours ago
- Business
- Miami Herald
Jaguar Health Provides Update on Meeting with FDA Discussing Statistically Significant Results of Responder Analysis of Breast Cancer Patients in Phase 3 OnTarget Trial and Potential Approval Pathway for Crofelemer
Productive and collaborative discussion on proposed pathway by the company to bring crofelemer to approval for cancer therapy-related diarrhea (CTD) in patients with metastatic breast cancer receiving selected targeted therapies The currently estimated US metastatic breast cancer population would qualify as an orphan population, which aligns with company's core focus on orphan diseases Company plans to promptly pursue authorization to initiate expanded access program for patients with breast cancer who may not be eligible for a potential pivotal treatment trial with crofelemer in patients with metastatic breast cancer SAN FRANCISCO, CA / ACCESS Newswire / June 9, 2025 / Jaguar Health, Inc.(NASDAQ:JAGX) (Jaguar) family company Napo Pharmaceuticals (Napo) today provided a recap on the company's assessment of the in-person Type C Meeting on May 28, 2025 with the Division of Gastroenterology of the U.S. Food and Drug Administration (FDA) to discuss the statistically significant responder analysis results for adult patients with breast cancer in Napo's recently conducted Phase 3 OnTarget trial. "We were very happy to take part in the face-to-face Type C Meeting, the catalyst for which was the positive data in the subpopulation of breast cancer patients from our OnTarget trial," said Lisa Conte, Jaguar's founder, president, and CEO. "Napo proposed two simultaneous potential pathways during the meeting for making crofelemer available to metastatic breast cancer patients with the significant unmet medical need of CTD: conducting a pivotal treatment trial to facilitate approval of crofelemer for CTD in this focused patient population; and the prompt pursuit of authorization to initiate an expanded access program for breast cancer patients with CTD who may not be eligible for this study, including breast cancer patients in the adjuvant and neoadjuvant settings. We are pleased with the constructive and productive discussion that took place with the FDA during the meeting. Last week the FDA formally acknowledged both of these key discussion points in correspondence to Napo. We plan to submit a protocol to the FDA for a pivotal treatment trial for a smaller number of metastatic breast cancer patients using crofelemer." Patient advocates participated in the Type C Meeting to share their raw and personal experience with CTD, including a metastatic breast cancer patient with uncontrollable diarrhea who received a prescription for crofelemer. "The design of the protocol for OnTarget was based on a survey of cancer patients. Today there are close to 100 approved targeted cancer agents. Thanks to these amazing drugs, metastatic cancer patients are living longer, frequently rendering cancer, and CTD, chronic ailments with which to live. To deepen our understanding of the current population of metastatic cancer breast with CTD, and ensure the clinical meaningfulness of the design of the anticipated pivotal treatment trial, we plan to conduct a new survey of this cancer patient population," Conte said. The currently estimated US metastatic breast cancer population potentially qualifies as an orphan population, in alignment with the company's core focus on orphan diseases. The company therefore intends to request orphan drug designation from the FDA for the CTD indication in this population. Given crofelemer's novel and paradigm-shifting mechanism of action, the company also plans to seek Breakthrough Therapy designation and/or Fast Track designation from the FDA to support potentially expedited regulatory approval in the US for crofelemer for CTD in metastatic breast cancer patients. "Embracing a sharp strategic focus on orphan indications seems to fit with the new administration's efforts and philosophy," said Conte. "Dr. Marty Makary, the new Commissioner of the FDA, commented in April 2025 that the agency will open a new regulatory pathway based on what he called a 'plausible mechanism,' focusing mainly on rare or incurable diseases." As expressed during the Type C Meeting, the data from the first-of-its-kind prophylactic OnTarget study is invaluable, providing new insights into the natural history of the important and debilitating side effect of CTD. Diarrhea is a common side effect of targeted cancer therapies and can lead to dose changes, treatment delays, and often cessation of lifesaving cancer therapy. The benefit to risk ratio of crofelemer is well-documented, as the active agent has been commercialized for the approved indication of HIV-related diarrhea since 2012, with no crofelemer-related reported serious adverse events. In both the US and European Union, crofelemer has been granted orphan drug designation for the orphan diseases of short bowel syndrome with intestinal failure and microvillus inclusion disease. Crofelemer has been granted orphan drug designation for treatment of diarrhea in cholera in the US, where cholera is an orphan disease. Orphan drug designation in the US qualifies the sponsor of a drug for various development incentives, including tax credits for qualified clinical testing and relief of filing fees. Additionally, orphan drug designation in the US provides a seven-year period of marketing exclusivity to the first sponsor who obtains marketing approval for the designated orphan drug. While the multicenter, double-blind, placebo-controlled OnTarget pivotal trial did not meet its primary endpoint, the subgroup of adult breast cancer patients achieved statistically significant results in the responder analysis. In the responder analysis of patients with breast cancer on targeted therapies, crofelemer CTD prophylaxis resulted in a greater proportion of monthly responders of diarrhea improvement compared to placebo. Patients with breast cancer accounted for 183 of the 287 participants in this unprecedented prophylactic clinical trial of crofelemer for diarrhea in adults with 10 solid tumor types receiving targeted therapy with or without standard chemotherapy. The OnTarget results in breast cancer patients were the subject of a poster presentation on December 11, 2024, at the San Antonio Breast Cancer Symposium, and additional significant results in adult breast cancer patients from the OnTarget study have been accepted for presentation as an oral rapid e-poster at the Multinational Association of Supportive Care in Cancer (MASCC) Annual Meeting in June 2025 in Seattle, Washington. About Crofelemer Crofelemer is a novel, oral plant-based prescription medicine purified from the red bark sap, also referred to as "dragon's blood," of the Croton lechleri tree in the Amazon Rainforest. Napo has established a sustainable harvesting program, under fair trade practices, for crofelemer to ensure a high degree of quality, ecological integrity, and support for indigenous communities. About the Jaguar Health Family of Companies Jaguar Health, Inc. (Jaguar) is a commercial stage pharmaceuticals company focused on developing novel proprietary prescription medicines sustainably derived from plants from rainforest areas for people and animals with gastrointestinal distress, specifically associated with overactive bowel, which includes symptoms such as chronic debilitating diarrhea, urgency, bowel incontinence, and cramping pain. Jaguar family company Napo Pharmaceuticals (Napo) focuses on developing and commercializing human prescription pharmaceuticals for essential supportive care and management of neglected gastrointestinal symptoms across multiple complicated disease states. Napo's crofelemer is FDA-approved under the brand name Mytesi® for the symptomatic relief of noninfectious diarrhea in adults with HIV/AIDS on antiretroviral therapy. Jaguar family company Napo Therapeutics is an Italian corporation Jaguar established in Milan, Italy in 2021 focused on expanding crofelemer access in Europe and specifically for orphan diseases. Jaguar Animal Health is a Jaguar tradename. Magdalena Biosciences, a joint venture formed by Jaguar and Filament Health Corp. that emerged from Jaguar's Entheogen Therapeutics Initiative (ETI), is focused on developing novel prescription medicines derived from plants for mental health indications. For more information about: Jaguar Health, visit Napo Pharmaceuticals, visit Napo Therapeutics, visit Magdalena Biosciences, visit Visit the Make Cancer Less Shitty patient advocacy program on Bluesky, X, Facebook & Instagram Forward-Looking Statements Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that the currently estimated US metastatic breast cancer population qualifies as an orphan population, Jaguar's expectation that the company will submit a protocol to the FDA for a pivotal treatment trial for a smaller number of metastatic breast cancer patients using crofelemer, Jaguar's expectation that it will promptly pursue authorization to initiate an expanded access program for patients with breast cancer who may not be eligible for a potential pivotal treatment trial with crofelemer in patients with metastatic breast cancer patients, and that the expanded access program would potentially include breast cancer patients in the adjuvant and neoadjuvant settings, Jaguar's expectation that Napo will conduct a clinical trial of crofelemer for treatment of CTD in patients with metastatic breast cancer, Jaguar's plans to seek Breakthrough Therapy designation and/or Fast Track designation from the FDA to support potentially expedited regulatory approval in the US for crofelemer for CTD in metastatic breast cancer patients, and Jaguar's expectation that additional significant results in adult breast cancer patients from the OnTarget study will be presented at the 2025 MASCC Annual Meeting. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "aim," "anticipate," "could," "intend," "target," "project," "contemplate," "believe," "estimate," "predict," "potential" or "continue" or the negative of these terms or other similar expressions. The forward-looking statements in this release are only predictions. Jaguar has based these forward-looking statements largely on its current expectations and projections about future events. These forward-looking statements speak only as of the date of this release and are subject to several risks, uncertainties, and assumptions, some of which cannot be predicted or quantified and some of which are beyond Jaguar's control. Except as required by applicable law, Jaguar does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Contact: hello@ Jaguar-JAGX SOURCE: Jaguar Health, Inc.
Indianapolis Star
a day ago
- Business
- Indianapolis Star
Jaguar Health Provides Update on Meeting with FDA Discussing Statistically Significant Results of Responder Analysis of Breast Cancer Patients in Phase 3 OnTarget Trial and Potential Approval Pathway for Crofelemer
Productive and collaborative discussion on proposed pathway by the company to bring crofelemer to approval for cancer therapy-related diarrhea (CTD) in patients with metastatic breast cancer receiving selected targeted therapies The currently estimated US metastatic breast cancer population would qualify as an orphan population, which aligns with company's core focus on orphan diseases Company plans to promptly pursue authorization to initiate expanded access program for patients with breast cancer who may not be eligible for a potential pivotal treatment trial with crofelemer in patients with metastatic breast cancer SAN FRANCISCO, CA / ACCESS Newswire / June 9, 2025 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) family company Napo Pharmaceuticals (Napo) today provided a recap on the company's assessment of the in-person Type C Meeting on May 28, 2025 with the Division of Gastroenterology of the U.S. Food and Drug Administration (FDA) to discuss the statistically significant responder analysis results for adult patients with breast cancer in Napo's recently conducted Phase 3 OnTarget trial. 'We were very happy to take part in the face-to-face Type C Meeting, the catalyst for which was the positive data in the subpopulation of breast cancer patients from our OnTarget trial,' said Lisa Conte, Jaguar's founder, president, and CEO. 'Napo proposed two simultaneous potential pathways during the meeting for making crofelemer available to metastatic breast cancer patients with the significant unmet medical need of CTD: conducting a pivotal treatment trial to facilitate approval of crofelemer for CTD in this focused patient population; and the prompt pursuit of authorization to initiate an expanded access program for breast cancer patients with CTD who may not be eligible for this study, including breast cancer patients in the adjuvant and neoadjuvant settings. We are pleased with the constructive and productive discussion that took place with the FDA during the meeting. Last week the FDA formally acknowledged both of these key discussion points in correspondence to Napo. We plan to submit a protocol to the FDA for a pivotal treatment trial for a smaller number of metastatic breast cancer patients using crofelemer.' Patient advocates participated in the Type C Meeting to share their raw and personal experience with CTD, including a metastatic breast cancer patient with uncontrollable diarrhea who received a prescription for crofelemer. 'The design of the protocol for OnTarget was based on a survey of cancer patients. Today there are close to 100 approved targeted cancer agents. Thanks to these amazing drugs, metastatic cancer patients are living longer, frequently rendering cancer, and CTD, chronic ailments with which to live. To deepen our understanding of the current population of metastatic cancer breast with CTD, and ensure the clinical meaningfulness of the design of the anticipated pivotal treatment trial, we plan to conduct a new survey of this cancer patient population,' Conte said. The currently estimated US metastatic breast cancer population potentially qualifies as an orphan population, in alignment with the company's core focus on orphan diseases. The company therefore intends to request orphan drug designation from the FDA for the CTD indication in this population. Given crofelemer's novel and paradigm-shifting mechanism of action, the company also plans to seek Breakthrough Therapy designation and/or Fast Track designation from the FDA to support potentially expedited regulatory approval in the US for crofelemer for CTD in metastatic breast cancer patients. 'Embracing a sharp strategic focus on orphan indications seems to fit with the new administration's efforts and philosophy,' said Conte. 'Dr. Marty Makary, the new Commissioner of the FDA, commented in April 2025 that the agency will open a new regulatory pathway based on what he called a 'plausible mechanism,' focusing mainly on rare or incurable diseases.' As expressed during the Type C Meeting, the data from the first-of-its-kind prophylactic OnTarget study is invaluable, providing new insights into the natural history of the important and debilitating side effect of CTD. Diarrhea is a common side effect of targeted cancer therapies and can lead to dose changes, treatment delays, and often cessation of lifesaving cancer therapy. The benefit to risk ratio of crofelemer is well-documented, as the active agent has been commercialized for the approved indication of HIV-related diarrhea since 2012, with no crofelemer-related reported serious adverse events. In both the US and European Union, crofelemer has been granted orphan drug designation for the orphan diseases of short bowel syndrome with intestinal failure and microvillus inclusion disease. Crofelemer has been granted orphan drug designation for treatment of diarrhea in cholera in the US, where cholera is an orphan disease. Orphan drug designation in the US qualifies the sponsor of a drug for various development incentives, including tax credits for qualified clinical testing and relief of filing fees. Additionally, orphan drug designation in the US provides a seven-year period of marketing exclusivity to the first sponsor who obtains marketing approval for the designated orphan drug. While the multicenter, double-blind, placebo-controlled OnTarget pivotal trial did not meet its primary endpoint, the subgroup of adult breast cancer patients achieved statistically significant results in the responder analysis. In the responder analysis of patients with breast cancer on targeted therapies, crofelemer CTD prophylaxis resulted in a greater proportion of monthly responders of diarrhea improvement compared to placebo. Patients with breast cancer accounted for 183 of the 287 participants in this unprecedented prophylactic clinical trial of crofelemer for diarrhea in adults with 10 solid tumor types receiving targeted therapy with or without standard chemotherapy. The OnTarget results in breast cancer patients were the subject of a poster presentation on December 11, 2024, at the San Antonio Breast Cancer Symposium, and additional significant results in adult breast cancer patients from the OnTarget study have been accepted for presentation as an oral rapid e-poster at the Multinational Association of Supportive Care in Cancer (MASCC) Annual Meeting in June 2025 in Seattle, Washington. About Crofelemer Crofelemer is a novel, oral plant-based prescription medicine purified from the red bark sap, also referred to as 'dragon's blood,' of the Croton lechleri tree in the Amazon Rainforest. Napo has established a sustainable harvesting program, under fair trade practices, for crofelemer to ensure a high degree of quality, ecological integrity, and support for indigenous communities. About the Jaguar Health Family of Companies Jaguar Health, Inc. (Jaguar) is a commercial stage pharmaceuticals company focused on developing novel proprietary prescription medicines sustainably derived from plants from rainforest areas for people and animals with gastrointestinal distress, specifically associated with overactive bowel, which includes symptoms such as chronic debilitating diarrhea, urgency, bowel incontinence, and cramping pain. Jaguar family company Napo Pharmaceuticals (Napo) focuses on developing and commercializing human prescription pharmaceuticals for essential supportive care and management of neglected gastrointestinal symptoms across multiple complicated disease states. Napo's crofelemer is FDA-approved under the brand name Mytesi ® for the symptomatic relief of noninfectious diarrhea in adults with HIV/AIDS on antiretroviral therapy. Jaguar family company Napo Therapeutics is an Italian corporation Jaguar established in Milan, Italy in 2021 focused on expanding crofelemer access in Europe and specifically for orphan diseases. Jaguar Animal Health is a Jaguar tradename. Magdalena Biosciences, a joint venture formed by Jaguar and Filament Health Corp. that emerged from Jaguar's Entheogen Therapeutics Initiative (ETI), is focused on developing novel prescription medicines derived from plants for mental health indications. For more information about: Jaguar Health, visit Napo Pharmaceuticals, visit Napo Therapeutics, visit Magdalena Biosciences, visit Visit the Make Cancer Less Shitty patient advocacy program on Bluesky, X, Facebook & Instagram Forward-Looking Statements Certain statements in this press release constitute 'forward-looking statements.' These include statements regarding Jaguar's expectation that the currently estimated US metastatic breast cancer population qualifies as an orphan population, Jaguar's expectation that the company will submit a protocol to the FDA for a pivotal treatment trial for a smaller number of metastatic breast cancer patients using crofelemer, Jaguar's expectation that it will promptly pursue authorization to initiate an expanded access program for patients with breast cancer who may not be eligible for a potential pivotal treatment trial with crofelemer in patients with metastatic breast cancer patients, and that the expanded access program would potentially include breast cancer patients in the adjuvant and neoadjuvant settings, Jaguar's expectation that Napo will conduct a clinical trial of crofelemer for treatment of CTD in patients with metastatic breast cancer, Jaguar's plans to seek Breakthrough Therapy designation and/or Fast Track designation from the FDA to support potentially expedited regulatory approval in the US for crofelemer for CTD in metastatic breast cancer patients, and Jaguar's expectation that additional significant results in adult breast cancer patients from the OnTarget study will be presented at the 2025 MASCC Annual Meeting. In some cases, you can identify forward-looking statements by terms such as 'may,' 'will,' 'should,' 'expect,' 'plan,' 'aim,' 'anticipate,' 'could,' 'intend,' 'target,' 'project,' 'contemplate,' 'believe,' 'estimate,' 'predict,' 'potential' or 'continue' or the negative of these terms or other similar expressions. The forward-looking statements in this release are only predictions. Jaguar has based these forward-looking statements largely on its current expectations and projections about future events. These forward-looking statements speak only as of the date of this release and are subject to several risks, uncertainties, and assumptions, some of which cannot be predicted or quantified and some of which are beyond Jaguar's control. Except as required by applicable law, Jaguar does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Contact: hello@ Jaguar-JAGX SOURCE: Jaguar Health, Inc. View the original press release on ACCESS Newswire
The Independent
23-05-2025
- Politics
- The Independent
Probation staff shortages threaten to derail plans to safely release prisoners early, ministers warned
Severe staff shortages mean the beleaguered probation service cannot safely monitor prisoners in the community under new plans to free up prison spaces, senior figures have warned. In a review ordered by Justice Secretary Shabana Mahmood as the prison crisis boiled over during her first days in office, her Tory predecessor, David Gauke advised freeing many prisoners a third of the way into their sentence and telling judges to avoid jailing people for less than a year in favour of community sentences. While the plans have been widely welcomed, concerns have been raised over how the under-pressure probation service will cope with an influx of new offenders to manage. Ms Mahmood is expected to accept most of the proposals, but former justice secretary Sir Robert Buckland expressed concern that the plans could merely 'transfer a prison-based problem into the community'. And probation union Napo warned that there was now a 'vital window' to invest in staff and their wellbeing before the changes place 'massive, massive pressure' on a service already in 'chaos'. Hailing the report as 'the most important review of sentencing policy in at least a generation', Napo chief Ian Lawrence added: 'But the problem the government has right now is, if prisons are full, so is the probation service. 'And our capacity to process even more people released into the community is going to be put under massive, massive pressure ... without something being done to maintain the confidence of staff. That means paying people so they don't just leave, because people [have had] enough of the current workload situation.' The most recent official figures suggest a shortfall of nearly 1,500 probation officers below the recommended staffing level of 7,115. Mr Lawrence warned that this target may underestimate the true need. Ministers have vowed to recruit a further 1,300 officers by March 2026 and while the probation service grew by 610 staff in the year to March, more than 2,000 staff – nearly 10 per cent – left over the same period. Forty per cent of probation officers who quit left with at least 10 years of experience, analysis by The Independent found. Asked whether he believes the probation service has enough staff to safely enact the reforms, Mr Lawrence said: 'Certainly not now.' Warning that probation officers 'are dealing with scores and scores – hundreds – of recall offenders every week, which is just stretching people's capacity to cope to the wire', he said the number of staff forced to take sickness days was 'very worrying'. More than 273,000 days were lost to sickness in the year to March, nearly a third more than in 2022, official figures show. With many regions majorly understaffed for years, HM chief inspector Martin Jones warned last year that 30 out of the 31 local probation units inspected in 2022-23 were judged to be either 'inadequate' or 'requiring improvement'. An ailing probation service can have fatal consequences, with major failings identified in the murders of Zara Aleena and a Terri Harris and her three children in Killamarsh the previous year. The Independent reported in November that the number of ex-offenders charged with committing serious crimes while on probation had surged by a third to hit a record high of 770 over the previous year, for crimes including murder, rape and serious violence. While he largely praised Mr Gauke's review, Tory former justice secretary Sir Robert Buckland warned that, unless backed up with more resources from the Treasury, 'I'm worried that we're going to transfer a prison-based problem into the community. Sir Robert said: 'If this is not properly resourced, my worry is that there would be supervision failures, further offences and inevitably that will cause massive public concern and political pressure to change course.' 'The one thing we need now is a very stable policy environment, so that the probation service can actually be resourced to get on with, under this system, an increasing role.' Also broadly backing the measures and praising Mr Gauke's assessment 'that we just cannot carry on as we are without doing something radical', former justice committee chair Sir Bob Neill warned that the £700m announced to bolster the service on Thursday 'can't be a one-off'. He added: 'The case that you'd make to Rachel Reeves is 'this is a classic case of investing to save', because ultimately much more spending on probation is going to be less than both the capital and revenue costs of much more prison places.' With much of those funds expected to be spent on a major extension of tagging and monitoring capabilities, Sir Bob commended Mr Gauke's call to 'toughen up community punishments as an alternative to prison'. But warning that probation has 'been a bit of Cinderella [service] for too long', Sir Bob said: 'There needs to be a proper workforce strategy for the probation service, a push on recruitment, better training and a concerted effort to raise the status of probation work.'
Miami Herald
20-05-2025
- Business
- Miami Herald
First Patient Randomized in Jaguar Health's Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial of Crofelemer in Pediatric Patients with Microvillus Inclusion Disease (MVID)
As announced, initial proof-of-concept (POC) results from distinct ongoing investigator-initiated trial (IIT) in Abu Dhabi show crofelemer reduced total parenteral support in a pediatric MVID patient with intestinal failure by up to 27%; per the IIT protocol, patient was taken off crofelemer after 12 weeks of treatment for a period of 30 days, but was restarted on daily crofelemer treatment after just 8 days, as patient's symptoms were worsening Additional POC IIT results expected throughout 2025 for MVID; results from company's MVID Phase 2 study expected with completion of treatment in H2 2026 Ground-breaking results in single-digit number of MVID patients could potentially lead to expedited approval in the EU through PRIME and to Breakthrough Therapy designation in the US SAN FRANCISCO, CA / ACCESS Newswire / May 20, 2025 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) family companies Napo Pharmaceuticals, Inc. (Napo) and Napo Therapeutics S.p.A. today announced that the first patient has been randomized in Napo's randomized, double-blind, placebo-controlled Phase 2 clinical trial to evaluate the safety and efficacy of the novel crofelemer oral powder for solution formulation in pediatric patients with intestinal failure due to the ultrarare disease MVID. Patients with MVID suffer from devastating diarrhea and dehydration caused by this debilitating, lifelong condition. These pediatric patients often require lifelong and life-sustaining total parenteral nutrition (TPN) with additional supplemental intravenous (IV) fluids, termed total parenteral support, which is the standard of care for MVID, for up to 7 days a week greater than 50% of the time each day to provide adequate nutrient and electrolyte support. While crucial for MVID patients, TPN carries a significant risk of morbidity and mortality due to infections, metabolic complications, liver and/or kidney disease, and a risk of neurodevelopmental delay. There are currently no approved drug treatments for MVID. As previously announced, and as presented last month at the 11th Annual ELITE PED-GI Congress in Abu Dhabi in the United Arab Emirates, the initial proof-of-concept results from the ongoing and independent proof-of-concept investigator-initiated trial (IIT) at Sheikh Khalifa Medical City (SKMC) showed that crofelemer reduced the required TPN and/or supplementary intravenous fluids - collectively referred to as parenteral support - in the first participating MVID patient by up to 27% at the end of 12-weeks of crofelemer treatment. In addition, this data showed that crofelemer reduced stool volume output and/or frequency of watery stools, and increased urine output - an indicator of improved nutrient oral absorption. Per the protocol for the IIT at SKMC, the pediatric MVID patient was taken off crofelemer after 12 weeks of treatment for a period of 30 days. After just 8 days, the patient's parents requested reinitiation of crofelemer dosing, as the patient's symptoms were worsening - evidenced by increased stool output and decreased urine output. Hence, the patient was restarted on daily treatment with crofelemer. "We're very happy that the first patient has been randomized in Napo's randomized, double-blind placebo-controlled Phase 2 clinical trial of crofelemer in pediatric MVID patients," said Lisa Conte, Jaguar's founder, president, and CEO. "This is another important milestone in development efforts for crofelemer for the treatment and management of intestinal failure related to this devastating ultrarare pediatric disease. For this study, randomization represents the key milestone of assigning a participating patient to either the crofelemer or placebo treatment group in a double-blind, placebo-controlled within-patient crossover study." "Additional proof-of-concept results for MVID from the ongoing IIT of crofelemer at SKMC in pediatric patients with intestinal failure due to MVID and other disorders are expected throughout 2025," Conte said. "The results of Napo's randomized double-blind, placebo-controlled Phase 2 study of crofelemer in pediatric MVID patients are expected to be available in the second half of 2026. Because the IIT of crofelemer for MVID is an unblinded, open-label study, results from this IIT are expected to be available sooner." Based on the initial proof-of-concept findings presented last month at the 11th Annual ELITE PED-GI Congress, crofelemer's novel antisecretory mechanism of action appears to have the potential to provide a novel therapeutic option to modify the disease progression associated with intestinal failure due to MVID in pediatric patients, as evidenced by the reductions in total parenteral support and potential to improve the quality of life of MVID patients. "Based on preliminary communications with European Medicines Agency (EMA) regulatory members that focus on PRIME (Priority Medicines), a program that provides enhanced interactions and early dialogue for development of novel medicines targeting unmet medical needs, we are excited by the initial findings from the IIT being conducted at SKMC," said Conte. "We are not aware of any other intervention that has shown the ability to reduce the required administration of life-sustaining parenteral support including TPN, thus potentially improving nutrient absorption in patients with MVID. Given the ultrarare nature of MVID, even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval in the European Union. Crofelemer may qualify for participation in PRIME in the European Union for MVID and potentially in the U.S. Food and Drug Administration's (FDA) Breakthrough Therapy program for this ultrarare pediatric indication. Additionally, in accordance with the guidelines of specific EU countries, published data from clinical investigations in MVID could support reimbursed early patient access to crofelemer for this debilitating condition." About Crofelemer Crofelemer is a novel, oral plant-based prescription medicine purified from the red bark sap, also referred to as "dragon's blood," of the Croton lechleri tree in the Amazon Rainforest. Napo has established a sustainable harvesting program, under fair trade practices, for crofelemer to ensure a high degree of quality, ecological integrity, and support for indigenous communities. About the Jaguar Health Family of Companies Jaguar Health, Inc. (Jaguar) is a commercial stage pharmaceuticals company focused on developing novel proprietary prescription medicines sustainably derived from plants from rainforest areas for people and animals with gastrointestinal distress, specifically associated with overactive bowel, which includes symptoms such as chronic debilitating diarrhea, urgency, bowel incontinence, and cramping pain. Jaguar family company Napo Pharmaceuticals (Napo) focuses on developing and commercializing human prescription pharmaceuticals for essential supportive care and management of neglected gastrointestinal symptoms across multiple complicated disease states. Napo's crofelemer is FDA-approved under the brand name Mytesi® for the symptomatic relief of noninfectious diarrhea in adults with HIV/AIDS on antiretroviral therapy. Jaguar family company Napo Therapeutics is an Italian corporation Jaguar established in Milan, Italy in 2021 focused on expanding crofelemer access in Europe and specifically for orphan and/or rare diseases. Jaguar Animal Health is a Jaguar tradename. Magdalena Biosciences, a joint venture formed by Jaguar and Filament Health Corp. that emerged from Jaguar's Entheogen Therapeutics Initiative (ETI), is focused on developing novel prescription medicines derived from plants for mental health indications. For more information about: Jaguar Health, visit Pharmaceuticals, visit Therapeutics, visit Biosciences, visit Visit the Make Cancer Less Shitty patient advocacy program on Bluesky, X, Facebook & Instagram Forward-Looking Statements Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that additional POC results for MVID from the IIT in pediatric patients with intestinal failure due to MVID and other disorders may be available throughout 2025, Jaguar's expectation that results from the company's MVID Phase 2 study will be available with completion of treatment in H2 2026, Jaguar's expectation that crofelemer's novel antisecretory mechanism of action may have the potential to provide a novel therapeutic option to modify the disease progression associated with intestinal failure due to MVID in pediatric patients and the potential to improve the quality of life of MVID patients, Jaguar's expectation that, given the ultrarare nature of MVID, even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval in the Europe Union, Jaguar's expectation that crofelemer may qualify for participation in PRIME in the European Union for MVID and potentially in the FDA's Breakthrough Therapyprogram, and Jaguar's expectation that, in accordance with the guidelines of specific EU countries, published data from clinical investigations in MVID could support reimbursed early patient access to crofelemer for this debilitating condition. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "aim," "anticipate," "could," "intend," "target," "project," "contemplate," "believe," "estimate," "predict," "potential" or "continue" or the negative of these terms or other similar expressions. The forward-looking statements in this release are only predictions. Jaguar has based these forward-looking statements largely on its current expectations and projections about future events. These forward-looking statements speak only as of the date of this release and are subject to several risks, uncertainties, and assumptions, some of which cannot be predicted or quantified and some of which are beyond Jaguar's control. Except as required by applicable law, Jaguar does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Contact: hello@ SOURCE: Jaguar Health, Inc.
Associated Press
20-05-2025
- Business
- Associated Press
First Patient Randomized in Jaguar Health's Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial of Crofelemer in Pediatric Patients with Microvillus Inclusion Disease (MVID)
As announced, initial proof-of-concept (POC) results from distinct ongoing investigator-initiated trial (IIT) in Abu Dhabi show crofelemer reduced total parenteral support in a pediatric MVID patient with intestinal failure by up to 27%; per the IIT protocol, patient was taken off crofelemer after 12 weeks of treatment for a period of 30 days, but was restarted on daily crofelemer treatment after just 8 days, as patient's symptoms were worsening Additional POC IIT results expected throughout 2025 for MVID; results from company's MVID Phase 2 study expected with completion of treatment in H2 2026 Ground-breaking results in single-digit number of MVID patients could potentially lead to expedited approval in the EU through PRIME and to Breakthrough Therapy designation in the US SAN FRANCISCO, CA / ACCESS Newswire / May 20, 2025 / Jaguar Health, Inc. ( NASDAQ:JAGX ) (Jaguar) family companies Napo Pharmaceuticals, Inc. (Napo) and Napo Therapeutics S.p.A. today announced that the first patient has been randomized in Napo's randomized, double-blind, placebo-controlled Phase 2 clinical trial to evaluate the safety and efficacy of the novel crofelemer oral powder for solution formulation in pediatric patients with intestinal failure due to the ultrarare disease MVID. Patients with MVID suffer from devastating diarrhea and dehydration caused by this debilitating, lifelong condition. These pediatric patients often require lifelong and life-sustaining total parenteral nutrition (TPN) with additional supplemental intravenous (IV) fluids, termed total parenteral support, which is the standard of care for MVID, for up to 7 days a week greater than 50% of the time each day to provide adequate nutrient and electrolyte support. While crucial for MVID patients, TPN carries a significant risk of morbidity and mortality due to infections, metabolic complications, liver and/or kidney disease, and a risk of neurodevelopmental delay. There are currently no approved drug treatments for MVID. As previously announced, and as presented last month at the 11th Annual ELITE PED-GI Congress in Abu Dhabi in the United Arab Emirates, the initial proof-of-concept results from the ongoing and independent proof-of-concept investigator-initiated trial (IIT) at Sheikh Khalifa Medical City (SKMC) showed that crofelemer reduced the required TPN and/or supplementary intravenous fluids - collectively referred to as parenteral support - in the first participating MVID patient by up to 27% at the end of 12-weeks of crofelemer treatment. In addition, this data showed that crofelemer reduced stool volume output and/or frequency of watery stools, and increased urine output - an indicator of improved nutrient oral absorption. Per the protocol for the IIT at SKMC, the pediatric MVID patient was taken off crofelemer after 12 weeks of treatment for a period of 30 days. After just 8 days, the patient's parents requested reinitiation of crofelemer dosing, as the patient's symptoms were worsening - evidenced by increased stool output and decreased urine output. Hence, the patient was restarted on daily treatment with crofelemer. 'We're very happy that the first patient has been randomized in Napo's randomized, double-blind placebo-controlled Phase 2 clinical trial of crofelemer in pediatric MVID patients,' said Lisa Conte, Jaguar's founder, president, and CEO. 'This is another important milestone in development efforts for crofelemer for the treatment and management of intestinal failure related to this devastating ultrarare pediatric disease. For this study, randomization represents the key milestone of assigning a participating patient to either the crofelemer or placebo treatment group in a double-blind, placebo-controlled within-patient crossover study.' 'Additional proof-of-concept results for MVID from the ongoing IIT of crofelemer at SKMC in pediatric patients with intestinal failure due to MVID and other disorders are expected throughout 2025,' Conte said. 'The results of Napo's randomized double-blind, placebo-controlled Phase 2 study of crofelemer in pediatric MVID patients are expected to be available in the second half of 2026. Because the IIT of crofelemer for MVID is an unblinded, open-label study, results from this IIT are expected to be available sooner.' Based on the initial proof-of-concept findings presented last month at the 11th Annual ELITE PED-GI Congress, crofelemer's novel antisecretory mechanism of action appears to have the potential to provide a novel therapeutic option to modify the disease progression associated with intestinal failure due to MVID in pediatric patients, as evidenced by the reductions in total parenteral support and potential to improve the quality of life of MVID patients. 'Based on preliminary communications with European Medicines Agency (EMA) regulatory members that focus on PRIME (Priority Medicines), a program that provides enhanced interactions and early dialogue for development of novel medicines targeting unmet medical needs, we are excited by the initial findings from the IIT being conducted at SKMC,' said Conte. 'We are not aware of any other intervention that has shown the ability to reduce the required administration of life-sustaining parenteral support including TPN, thus potentially improving nutrient absorption in patients with MVID. Given the ultrarare nature of MVID, even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval in the European Union. Crofelemer may qualify for participation in PRIME in the European Union for MVID and potentially in the U.S. Food and Drug Administration's (FDA) Breakthrough Therapy program for this ultrarare pediatric indication. Additionally, in accordance with the guidelines of specific EU countries, published data from clinical investigations in MVID could support reimbursed early patient access to crofelemer for this debilitating condition.' About Crofelemer Crofelemer is a novel, oral plant-based prescription medicine purified from the red bark sap, also referred to as 'dragon's blood,' of the Croton lechleri tree in the Amazon Rainforest. Napo has established a sustainable harvesting program, under fair trade practices, for crofelemer to ensure a high degree of quality, ecological integrity, and support for indigenous communities. About the Jaguar Health Family of Companies Jaguar Health, Inc. (Jaguar) is a commercial stage pharmaceuticals company focused on developing novel proprietary prescription medicines sustainably derived from plants from rainforest areas for people and animals with gastrointestinal distress, specifically associated with overactive bowel, which includes symptoms such as chronic debilitating diarrhea, urgency, bowel incontinence, and cramping pain. Jaguar family company Napo Pharmaceuticals (Napo) focuses on developing and commercializing human prescription pharmaceuticals for essential supportive care and management of neglected gastrointestinal symptoms across multiple complicated disease states. Napo's crofelemer is FDA-approved under the brand name Mytesi® for the symptomatic relief of noninfectious diarrhea in adults with HIV/AIDS on antiretroviral therapy. Jaguar family company Napo Therapeutics is an Italian corporation Jaguar established in Milan, Italy in 2021 focused on expanding crofelemer access in Europe and specifically for orphan and/or rare diseases. Jaguar Animal Health is a Jaguar tradename. Magdalena Biosciences, a joint venture formed by Jaguar and Filament Health Corp. that emerged from Jaguar's Entheogen Therapeutics Initiative (ETI), is focused on developing novel prescription medicines derived from plants for mental health indications. For more information about: Jaguar Health, visit Napo Pharmaceuticals, visit Napo Therapeutics, visit Magdalena Biosciences, visit Visit the Make Cancer Less Shitty patient advocacy program on Bluesky, X, Facebook & Instagram Forward-Looking Statements Certain statements in this press release constitute 'forward-looking statements.' These include statements regarding Jaguar's expectation that additional POC results for MVID from the IIT in pediatric patients with intestinal failure due to MVID and other disorders may be available throughout 2025, Jaguar's expectation that results from the company's MVID Phase 2 study will be available with completion of treatment in H2 2026, Jaguar's expectation that crofelemer's novel antisecretory mechanism of action may have the potential to provide a novel therapeutic option to modify the disease progression associated with intestinal failure due to MVID in pediatric patients and the potential to improve the quality of life of MVID patients, Jaguar's expectation that, given the ultrarare nature of MVID, even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval in the Europe Union, Jaguar's expectation that crofelemer may qualify for participation in PRIME in the European Union for MVID and potentially in the FDA's Breakthrough Therapyprogram, and Jaguar's expectation that, in accordance with the guidelines of specific EU countries, published data from clinical investigations in MVID could support reimbursed early patient access to crofelemer for this debilitating condition. In some cases, you can identify forward-looking statements by terms such as 'may,' 'will,' 'should,' 'expect,' 'plan,' 'aim,' 'anticipate,' 'could,' 'intend,' 'target,' 'project,' 'contemplate,' 'believe,' 'estimate,' 'predict,' 'potential' or 'continue' or the negative of these terms or other similar expressions. The forward-looking statements in this release are only predictions. Jaguar has based these forward-looking statements largely on its current expectations and projections about future events. These forward-looking statements speak only as of the date of this release and are subject to several risks, uncertainties, and assumptions, some of which cannot be predicted or quantified and some of which are beyond Jaguar's control. Except as required by applicable law, Jaguar does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Contact: [email protected] Jaguar-JAGX SOURCE: Jaguar Health, Inc. press release
